Modification of epor-encoding nucleic acids
AssigneeEnsoma, Inc.
InventorsAshvin Reddy Bashyam, Martha Ann Clark, Pak Wai Au, Kush M. Parmar
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Abstract
Gene therapy typically modifies some, but not all, cells of a population of target cells. One approach to increasing the prevalence of modified cells includes delivering to the modified cells a gene that provides a competitive advantage (e.g., a proliferative advantage) and therefore results in enrichment of modified cells. The present disclosure including, among other things, methods and compositions for providing a competitive advantage to one or more cells by providing to the cells a nucleic acid encoding a signaling-enhanced EpoR polypeptide (e.g., a truncated EpoR or gain-of-function EpoR).
Claims69
A method comprising contacting one or more cells of a mammalian subject with (i) an editing enzyme that modifies an endogenous erythropoietin receptor (EpoR)-encoding nucleic acid to produce a modified EpoR-encoding nucleic acid, wherein the modified EpoR-encoding nucleic acid encodes a signaling-enhanced EpoR polypeptide, or (ii) a transgene that encodes a signaling-enhanced EpoR polypeptide,
- wherein the contacting occurs in vivo, in vitro, or ex vivo.
Use of (i) an editing enzyme for modification of an endogenous erythropoietin receptor (EpoR)-encoding nucleic acid of one or more cells of a mammalian subject to produce a modified EpoR-encoding nucleic acid, wherein the modified EpoR-encoding nucleic acid encodes a signaling-enhanced EpoR polypeptide or (ii) a transgene that encodes a signaling-enhanced EpoR polypeptide,
- wherein the use comprises contacting one or more cells of a mammalian subject with the editing enzyme or transgene in vivo, in vitro, or ex vivo.
A nucleic acid encoding (i) an editing enzyme that modifies an endogenous erythropoietin receptor (EpoR)-encoding nucleic acid to produce a modified EpoR-encoding nucleic acid, wherein the modified EpoR-encoding nucleic acid encodes a signaling-enhanced EpoR polypeptide, and optionally a guide RNA, or (ii) a transgene that encodes a signaling-enhanced EpoR polypeptide, and optionally (iii) a nucleic acid sequence encoding a therapeutic agent.
Dependencies are inferred from the claim text.